Last reviewed 2026-04-20 · How we verify

Rhgalns (ELOSULFASE ALFA)

VIMIZIM provides exogenous N-acetylgalactosamine-6-sulfatase to increase the catabolism of GAGs KS and C6S in lysosomes.

At a glance

Mechanism of action

MPS IVA is caused by a deficiency in N-acetylgalactosamine-6-sulfatase, leading to the buildup of GAGs KS and C6S in lysosomes. VIMIZIM supplies this enzyme to help break down these GAGs, reducing cellular dysfunction.

Approved indications

• Mucopolysaccharidosis, MPS-IV-A

Boxed warnings

• WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS and RISK OF ACUTE RESPIRATORY COMPLICATIONS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate VIMIZIM in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue VIMIZIM and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1) ] . Patients with acute respiratory illness may be at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions and require additional monitoring [see Warnings and Precautions (5.2) ] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS and RISK OF ACUTE RESPIRATORY COMPLICATIONS See full prescribing information for complete boxed warning . Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. ( 5.1 ) Initiate VIMIZIM in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) If a severe hypersensitivity reaction (anaphylaxis) occurs, discontinue VIMIZIM and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 ) Patients with acute respiratory illness may be at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions, and require additional monitoring ( 5.2 )

Common side effects

• pyrexia
• vomiting
• headache
• nausea
• abdominal pain
• chills
• fatigue

Key clinical trials

• PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) (PHASE1)
• Natural History of Atypical Morquio A Disease
• A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
• BMN 110 Phase 3B in Australian Patients (PHASE3)
• Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome (PHASE2)
• Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation (PHASE2)
• A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) (PHASE3)

Primary sources

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